作者: H. Trent Spencer , Jacques Galipeau
DOI: 10.1007/978-1-59259-734-5_27
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摘要: Since the initial confirmation that genetic alterations cause disease, pervasive outlook has been future for gene therapy is very promising, but curing diseases by transfer of specific nucleic acid sequences problematic. It should be recognized, however, hurdles facing investigators in field can defined and are likely not insurmountable. The major limitations inability to genetically modify sufficient numbers target cells express transferred gene(s) over prolonged periods (1,2). With respect application treatment cancer, many aspects fundamental these different than those faced pioneers other treatments, example, development chemotherapy agents, which mainly a lack understanding biological systems being manipulated. Even so, great strides accomplishments have achieved during last decade, only with physical genes into also clinical practice (3). purpose this chapter provide general background on how as well example focusing translation study from inception through completed Phase III trial.